Artegene

Artegene (also known as G2ARTE-transduced autologous CD34+ cells) is an investigational ex vivo gene therapy developed for the treatment of Artemis-deficient severe combined immunodeficiency (DCLRE1C-SCID). The therapy involves the collection of a patient's own CD34+ hematopoietic stem and progenitor cells, which are then transduced ex vivo with the G2ARTE lentiviral vector. This vector delivers a functional copy of the human DCLRE1C cDNA under the control of its endogenous promoter to restore the production of the Artemis protein. Artemis is a critical endonuclease required for V(D)J recombination during T and B lymphocyte development; its deficiency results in a complete lack of adaptive immunity and increased cellular sensitivity to ionizing radiation. Once the modified cells are re-infused into the patient, they engraft in the bone marrow and give rise to a functional immune system.

Other names

G2ARTE-transduced autologous CD34+ cellsDCLRE1C gene therapyDCLRE-1C gene therapyDCLRE 1C gene therapyArtemis-SCID gene therapy